Rare Disease

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SMA spinal muscular atrophy | Image Credit: © Rochu_2008 - stock.adobe.com

Protein Level Correlates With Pediatric SMA Severity, Treatment Response

By Jared Kaltwasser

October 17th 2024

Investigators say thrombospondin-4 levels were reduced in patients with symptoms of spinal muscular atrophy (SMA), but levels of the protein increased after therapy.

SMA puzzle pieces | image credit: Dzmitry - stock.adobe.com

Muscle-Directed Therapy Apitegromab Meets Primary End Point in Phase 3 SAPPHIRE Trial of SMA

By Marco Meglio

October 17th 2024

Child at pediatrician's office holding teddy bear | Image credit: VadimGuzhva – stock.adobe.com

Substantial Delays in Postapproval Pediatric Testing for New Drugs

By Hayden E. Klein

October 7th 2024

Newborn screening can provide numerous benefits for infants who may have unknown conditions or complications | image credit: Peakstock - stock.adobe.com

5-Year Review Further Bolsters Support for Newborn SMA Screening

October 3rd 2024

SMA has previously been associated with multiple metabolic and nutritional issues | image credit: JEGAS RA - stock.adobe.com

New Research Unearths Evidence of Global Metabolic Disruption in Patients With SMA

September 21st 2024

Exploring the Implementation of Quality Care Programs for Patients with Myeloproliferative Neoplasms

Bruce Feinberg, DO; Kathy Oubre, Michael Reff, RPh, MBA; Jamile M. Shammo, MD; and Ruben Mesa, MD, discuss the incorporation of quality care programs for improved disease management and patient care for MPNs.

Exploring the Implementation of Quality Care Programs for Patients with Myeloproliferative Neoplasms peer exchange

More News

September 1st 2017

5 Significant Developments With CAR-T Therapy

Surabhi Dangi-Garimella, PhD

A look at some of the major developments in the chimeric antigen receptor T (CAR-T) cell space.

September 1st 2017

What We're Reading: HHS Cuts ACA Ads; Chemical Hazard in Houston; CAR-T Treatment Price Tag

August 31st 2017

Abeona Receives Breakthrough Designation for Rare Skin Disease Gene Therapy

A gene therapy for patients with recessive dystrophic epidermolysis bullosa (RDEB), a rare skin disease that causes the skin to be very fragile and blister easily, has been granted Breakthrough Therapy designation status by the FDA.

August 30th 2017

FDA Approves Tisagenlecleucel, the First CAR-T Cell Therapy in the United States

Laura Joszt, MA

The FDA has approved the first chimeric antigen receptor-T (CAR-T) treatment, tisagenlecleucel (Kymriah), for the treatment of B-cell precursor acute lymphoblastic leukemia in certain pediatric and young adult patients. The therapy represents a new frontier in cancer care.

August 25th 2017

Case Study Identifies New Trigger for CAR-T Cell Expansion in DLBCL in JCAR017 Trial

Surabhi Dangi-Garimella, PhD

Chimeric antigen receptor (CAR)-T cells can re-expand in a lymphoma patient months after the initial infusion and can also be active against the patient’s cancer, according to a new study.

August 21st 2017

FDA Approves Besponsa for Relapsed/Refractory Acute Lymphoblastic Leukemia

Alison Rodriguez

The FDA has approved Besponsa (inotuzumab ozogamicin) for treating adults with relapsed or refractory B-cell precursor acute lymphoblastic leukemia.

August 18th 2017

What We're Reading: Mylan Settlement; MS Pricing Probe; Iowa Insurer's Premium Rate Hike

August 18th 2017

Understanding Patient Preferences Can Promote Adherence in MS Patients, Study Says

Alison Rodriguez

Patient preferences for multiple sclerosis therapies are important to take into account and are increasingly so as alternatives to traditional injection therapies are developed to address adherence issues.

August 10th 2017

ICER Seeks Public Comment on Scoping Document for CAR-T Treatments

Surabhi Dangi-Garimella, PhD

The Institute for Clinical and Economic Review (ICER) has released a draft version of their scoping document that will compare the clinical effectiveness and value of 2 chimeric antigen receptor (CAR)-T cell treatments being reviewed by the FDA.

August 10th 2017

Vaccine for Glioblastoma Awarded Orphan Drug Status by FDA

Christina Mattina

MimiVax announced that its immunotherapy vaccine SurVaxM, which is being tested in clinical trials for glioblastoma, has been designated an orphan drug by the FDA.

August 9th 2017

Results of Phase 2 Study Investigating Luspatercept in MDS

Alison Rodriguez

The results of a phase 2 study that explored the effects of luspatercept in patients with lower-risk myelodysplastic syndromes (MDS) were presented at the 22nd Congress of the European Hematology Association by Acceleron Pharma Inc. The company is developing the drug with Celgene.

August 7th 2017

NCI Researchers Discuss Prospects and Challenges of Treating Glioblastoma

Christina Mattina

Amidst news of Senator John McCain’s glioblastoma diagnosis, a recent discussion between 2 neuro-oncology researchers explored the reasons why this aggressive brain tumor is so difficult to manage, and outlined the ongoing investigations into possible therapies.

August 6th 2017

Study Examines Inpatient Rehabilitation for Patients With Marfan Syndrome

Alison Rodriguez

Patients with congenital heart defects, such as Marfan syndrome (MFS), are experiencing longer life expectancies due to new advances in medicine-resulting in a greater need for medical rehabilitation.

August 5th 2017

Phase 1 and Phase 2 Study Results Find Potential Treatment for Severe Cystic Fibrosis

Alison Rodriguez

Vertex Pharmaceuticals recently announced positive data from phase 1 and 2 studies of 3 triple combination regimens in cystic fibrosis patients with one F508del mutation and one minimal function mutation (F508del/Min). These results are the first to present the potential of treating the underlying cause of those with difficult-to-treat cystic fibrosis.

August 4th 2017

5 Developments With Cancer Immunotherapy

Surabhi Dangi-Garimella, PhD

There’s a lot happening in clinical practice with immunotherapy treatments: expanded indications for nivolumab and ipilimumab, biomarkers to judge patient response to combinations, and developments with CAR-T.

This Week in Managed Care: August 4, 2017

August 4th 2017

This Week in Managed Care: August 4, 2017

This week, the top managed care stories included bipartisan proposals for fixing the Affordable Care Act (ACA); FDA approved a targeted therapy for patients with acute myeloid leukemia who have a rare genetic mutation; and a study found that more patients have gained access to clinical trials after the ACA was implemented.

August 2nd 2017

ICER Seeks Comments for Proposed Changes for Orphan Drugs Assessments

Alison Rodriguez

The Institute for Clinical and Economic Review is seeking public comments on its recently released outline of proposed adaptations involving the assessment of certain treatments for ultra-rare conditions, also known as orphan drugs.

August 2nd 2017

CAR-T Cells Can Increase Clinical Remission Rates in Multiple Myeloma Patients

Alison Rodriguez

The new immunotherapy of chimeric antigen receptor (CAR)-T cells has demonstrated the ability to increase clinical remission in multiple myeloma patients by targeting the B-cell maturation protein that participates in disease progression.

August 1st 2017

Enasidenib Approved for IDH2-Mutated Patients With Relapsed/Refractory AML

Surabhi Dangi-Garimella, PhD

Enasidenib (Idhifa), developed by Celgene and Agios, has been granted FDA approval in tandem with a companion diagnostic test to detect IDH-2 mutations.

July 30th 2017

FDA Expands Approval of Yervoy for Metastatic Melanoma in Pediatric Patients

Alison Rodriguez

The FDA has expanded the indication of Bristol-Myers Squibb’s Yervoy (ipilimumab) to include the treatment of pediatric patients 12 years of age and older with unresectable or metastatic melanoma.

July 28th 2017

Breakthrough Therapy Designation for Venetoclax in AML

Venetoclax (Venclexta), in combination with low dose cytarabine, has been granted a breakthrough therapy designation for use in elderly, treatment-naive patients with acute myeloid leukemia (AML) who cannot handle intensive chemotherapy.

July 26th 2017

Patient Questionnaires Can Predict Clinical Outcomes in Patients With Multiple Sclerosis

Alison Rodriguez

Patient-reported outcomes (PROs) can demonstrate the effects of a disease or treatment. By using patient questionnaires, the results can be analyzed to predict clinical outcomes in medical research for diseases, including multiple sclerosis (MS).

July 25th 2017

Mechanism for Checkpoint Inhibitor Resistance Identified in Invasive Bladder Cancer

Christina Mattina

Researchers have identified a cellular signaling pathway that when activated by mutations may prevent immune cells from infiltrating bladder tumors, thus making the cancer resistant to immune checkpoint inhibitors.

July 25th 2017

What We're Reading: Monthly HIV Shot; Molina Layoffs; Charlie Gard Treatment

July 20th 2017

Rare Diseases Create Significant Care and Financial Burdens Among Patients

July 19th 2017

Nivolumab, Alone or With Ipilimumab, Met the Disease Control Rate in Malignant Pleural Mesothelioma

In a late-breaking abstract at the 2017 American Society of Clinical Oncology Annual Meeting, researchers presented an interim analysis of a randomized phase 2 trial of second- or third-line nivolumab, with or without ipilimumab, in patients with second- or third-line malignant pleural mesothelioma.

July 18th 2017

New Report Examines the Biopharmaceutical Pipeline and Innovation in Clinical Development

Alison Rodriguez

Report notes that 74% of drugs that are currently in development are potentially first-in-class medicines, and 822 drugs in development are for rare diseases.

July 17th 2017

Daratumumab-Based Combination Effective in Relapsed/Refractory Multiple Myeloma, Short PFS in Heavily Pretreated Patients

Surabhi Dangi-Garimella, PhD

Managing heavily pretreated, often less fit, patients with relapsed/refractory multiple myeloma is a challenge in routine practice, as illustrated by the fact progression-free survival remains short, although daratumumab-based combination therapies are proven effective, according to results presented during a poster session at the 2017 American Society of Clinical Oncology Annual Meeting.

July 17th 2017

Phase 1 Study Results at ASCO Support First-Line Use of Daratumumab in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

A phase 1 study, presented at the 2017 Annual Meeting of the American Society of Clinical Oncology, found that using daratumumab, an antibody that binds and inhibits the CD38 receptor, can improve patient response to treatment.

July 17th 2017

Daratumumab With Standard Regimen Improves PFS, ORR Independent of Cytogenetic Risk in Multiple Myeloma

Surabhi Dangi-Garimella, PhD

Updated trial results at the 2017 Annual Meeting of the American Society of Clinical Oncology showed the combinations of daratumumab with either lenalidomide and dexamethasone or bortezomib and dexamethasone prolonged progression-free survival for patients with relapsed refractory multiple myeloma.

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